Takeda reconsiders future of therapy for rare genetic condition following PhII fail

Takeda’s Phase II program in a rare hereditary disease called metachromatic leukodystrophy (MLD) is “likely to be discontinued” after failing its primary and secondary endpoints, the company revealed Thursday in its latest quarterly earnings report.

MLD is a fast-progressing and fatal neurodegenerative condition that can affect patients’ movement, speech, ability to walk, and even behavior. It’s commonly diagnosed in childhood, and caused by the deficiency of an enzyme called arylsulfatase A. Takeda’s candidate, TAK-611, is an enzyme replacement therapy that makes use of technology licensed from BioMarin. However, the company noted Thursday that the candidate “did not meet primary and secondary endpoints” in a Phase II study.

Andy Plump

“We are disappointed and wish to express our gratitude to the MLD children, parents and caregivers. We’re currently evaluating options. But given these outcomes, the program unfortunately is likely to be discontinued,” R&D president Andy Plump said on Takeda’s Q1 call with investors, according to an AlphaSense transcript.

The R&D team is also reconsidering the future of its EGFR inhibitor Exkivity as a monotherapy for certain first-line non-small cell lung cancer patients. The drug is currently approved for NSCLC patients with EGFR exon 20 insertion mutations who have progressed on or after platinum-based chemotherapy. However, a Phase III trial testing it in the first-line setting was “stopped for futility,” according to Plump. Takeda plans on discussing with regulators and “determining next steps for the program,” he said.

“While we are disappointed with this outcome, we are continuing to evaluate the full data set to gain a greater understanding of the findings. We will provide an update when available,” a Takeda spokesperson told Endpoints News on Thursday.

Costa Saroukos

CFO Costa Saroukos cited Takeda’s “growth and launch” products as drivers of revenue growth in the first quarter, which now includes dengue vaccine Qdenga. Takeda scored its first approval for TAK-003 in 2022 in Indonesia, and has since picked up a suite of approvals in the EU, United Kingdom, Brazil, Argentina, and Thailand. However, the company yanked its application in the US a couple weeks ago after regulators “sought additional data not captured within the TAK-003 clinical trial protocol,” which the agency had previously reviewed and accepted.

The company reaffirmed on Thursday that its plan in the US will be “further evaluated given the need for travelers and those living in dengue-endemic areas of the U.S., such as Puerto Rico.”

Meanwhile, CEO Christophe Weber said on the Q1 call that Takeda is “encouraged by the positive momentum” in their early launch process in other countries, and is “already seeing early signs of higher-than-expected demand.”

Due to high demand, Takeda is ramping up its manufacturing efforts, and last week opened a new facility for drug substance production in Germany. By the end of the decade, the company hopes to achieve an annual supply capacity of about 100 million doses, and it’s currently exploring partnerships to help get there, particularly with manufacturers in endemic countries.

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